PB2672: EXPLORING UNMET NEEDS OF PATIENTS WITH MYELODYSPLASTIC NEOPLASMS AND CAREGIVERS IN A NATIONAL ITALIAN SURVEY
Authors: Elena Crisà, Annamaria Nosari, Daniela Cilloni, Sam Salek, Tatyana Ionova, Matteo Giovanni Della Porta, Maria Teresa Voso, Luca Maurillo, Carlo Finelli, Valeria Santini, Enrico Balleari, Federica Pilo, Giuseppe Palumbo, Alfredo Molteni, Marta Riva, Esther Oliva
Journal: HemaSphere (2023)

Topic: 35. Quality of life and palliative care Background: One of the main goals of treatment for patients with myelodysplastic neoplasms (MDS) is maintaining/improving quality of life (QoL). It is known that transfusion dependence impacts QoL due to persistent severe anemia but also due to the burden of the frequency and time spent in hospital, the inability to travel and the dependence on hospital and staff, amongst other factors. Aims: We performed a survey to explore the impact of MDS and treatment on patients and caregivers, patients’ preferences and their unmet needs in Italy. Methods: This ongoing survey (online and paper versions) is being promoted by Associazione Italiana Pazienti Sindromi Mielodisplastica (AIPaSiM) advocacy group. Sixty items relevant to patients and 20 relevant to caregivers were identified by hematologists and patients. The patient-reported outcome measures (PROMs), QOL-E and HM-PRO, are included in the survey. Lower scores in QOL-E reflect better outcomes while higher scores in HM-PRO represent worse outcomes. Results: From June 2022, 179 patients (median age 73 years, females 51%) and 46 caregivers (76% females, mostly family members) completed the survey. QOL-E and HM-PRO scores are reported in Table 1. Seventy-seven percent of patients were retired. Thirty-one percent of patients was diagnosed more than 5 years before the survey and 67% were receiving treatment: 40% for lower risk and 25% for higher risk MDS (2% unknown). Twenty-six percent lived far from the treating center and, for 47%, travelling to hospital was claimed distressful and, with respect to those that were not disturbed by travel, was associated with significantly worse median HM-PRO physical (53 vs 28, p<0.001), emotional (54 vs 36, p=0.005), social (33 vs 0, p=0.003), eating and drinking habits (50 vs 0, p=0.001), Part A impact score (43 vs 23, p<0.001) and Part B symptom score (27 vs 18, p=0.002) and QOL-E functional (11 vs 42, p<0.001), social (12 vs 31, p<0.001)), fatigue (30 vs 38, p=0.001), MDS specific (23 vs 34, p=0.001), general scores (22 vs 31, p=0.002), treatment-outcome index (21 vs 29, p=0.001). Sixty-seven patients received transfusions. The increasing frequency of transfusions (26% low; 61% intermediate, 26% high) was associated with worse HM-PRO physical (p=0.002), social (p=0.002), and symptoms scores (p=0.042) and QOL-E functional (p=0.008), social (p=0.011), fatigue (p= 0.016), MDS-specific (p<0.001), general (p=0.006) and treatment-outcome index (p=0.001) scores. Only 2% received transfusions at home; however, 40% percent would prefer being transfused at home (30% of those treated in the center of care and 50% of those treated in another center) and such patients had worse QOL-E functional (p=0.002), fatigue (p=0.02), treatment-outcome index(p= 0.012) scores, as well as worse HM-PRO physical behavior (p=0.001), emotional behavior (p=0.013) and part A impact (p=0.004) scores. For 40% of patients, waiting times in the hospital was associated with a significant negative impact (HM-PRO part A impact score p=0.012). Other unmet needs were comprehension of disease and treatment options at diagnosis (for 40% and 24%, respectively) and access to new drugs. The importance of the role of the caregiver is highlighted by the fact that 50% of patients go to hospital with the caregiver. Caregivers’ work-life was affected for 37%, leading to a job change in 43% of cases. Summary/Conclusion: Understanding patients’ unmet needs and their impact on their quality of life and symptoms is crucial to providing optimized patient care and guide haematologists and governance choices.Keywords: Quality of life, Clinical data, MDS, Health care